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Alnylam, Tekmira target RNAi therapeutics
September 2009
by Lloyd Dunlap  |  Email the author


CAMBRIDGE, Mass.—In a move aimed at the discovery of novel cationic lipids and lipid nanoparticles for the systemic delivery of RNAi therapeutics, Alnylam Pharmaceuticals Inc. and Tekmira Pharmaceuticals Corp. have joined forces and launched a new company, AlCana Technologies Inc.  
The research collaboration will be funded by Alnylam and conducted by AlCana, a company founded by former Tekmira scientists in association with lipid experts in Peter Cullis' lab at the University of British Columbia, which will also participate in the research collaboration.  
"Strategically," notes Barry Greene, president and COO of Alnylam, "continuously improving delivery is a critical area, and with this collaboration and others we are on a clear trajectory to achieve low, single-digit microgram per kilogram efficacy with lipid nanoparticles."  
Lipid nanoparticle technology for the delivery of RNAi drugs is the most advanced delivery technology in clinical development, says Tekmira CEO Mark Murray.
"Tekmira's SNALP (stable nucleic acid-lipid particle) technology has been shown in preclinical models to be a safe and effective way to deliver RNAi drugs to disease sites. Cationic lipids are one component of Tekmira's SNALP nanoparticle technology. By changing the components and ratios of the different SNALP components, including cationic lipids, Tekmira can adjust the properties of a given formulation for delivery of RNAi drugs to different organs and tissues in the body," he states.
Under the terms of the two-year research collaboration, Alnylam will receive exclusive rights to all new inventions as well as rights to sublicense any resulting intellectual property to Alnylam's current and future partners. Tekmira receives rights to use new inventions for their own RNAi therapeutic programs licensed under Alnylam intellectual property through its InterfeRx program. AlCana will focus its efforts on generating novel cationic lipids that can be incorporated into lipid nanoparticles for the delivery of RNAi drugs.  
In March 2008, Alnylam granted Tekmira InterfeRx licenses to discover, develop and commercialize RNAi therapeutics towards seven gene targets—one of which is apolipoprotein B (ApoB). Tekmira has initiated a Phase I human clinical trial for ApoB SNALP, which is being developed as a treatment for patients with high LDL cholesterol, or "bad" cholesterol, and expects to complete the Phase I trial in early 2010. Alnylam is eligible to receive milestones and royalties based on the development and commercialization of the program. 
Alnylam also has a program in Phase I trials formulated using Tekmira's SNALP technology, Greene notes. Called ALN-VSP, the proprietary Alnylam program is being developed for the treatment of liver cancers and potentially other solid tumors. ALN-VSP targets two key genes involved in the disease pathway of liver cancer: KSP (kinesin spindle protein), which is involved in cancer proliferation, and VEGF (vascular endothelial growth factor), which is involved in cancer angiogenesis. In April 2009, ALN-VSP entered into a Phase I multi-center, open label, dose escalation study for patients around the age of 55 with advanced solid tumors with liver involvement, who have failed to respond to or have progressed after standard treatment. 
"We expect to complete enrollment and present preliminary data from this study in mid-2010," Greene states.
Tekmira is eligible to receive milestones and royalties based on the development and commercialization of any Alnylam program that uses Tekmira's proprietary technologies, including SNALP.  
RNAi drugs act to target the root cause of disease by silencing genes associated with disease, thereby acting upstream of today's therapies. The commercial potential will be driven by the specific indications treated by the RNAi drugs, Murray says.
"It is too early to be more specific than that," he states. "We believe RNAi represents a breakthrough in biology that has the opportunity to treat diseases in a fundamentally new way by targeting virtually any gene in the genome involved in the causal pathway of disease, including genes that are currently 'undrugable' using today's medicines," Greene adds.  
Alnylam is developing RNAi therapeutics for the treatment of a wide range of diseases, including liver cancers, hypercholesterolemia, Huntington's disease and TTR amyloidosis. The company has alliances with Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin and Cubist.    

Alnylam and Regulus receive U.S. patent covering microRNA therapeutics
CAMBRIDGE, Mass.—Alnylam Pharmaceuticals Inc. and Regulus Therapeutics Inc. announced Aug. 11 that the U.S. Patent and Trademark Office (USPTO) awarded a notice of allowance for the Manoharan patent (Application No. 11/200,703), which covers several chemical classes of anti-miRs, antisense oligonucleotide inhibitors of microRNAs. This new patent in combination with Regulus' existing intellectual property estate broadly covers approaches to developing microRNA-based therapeutics.
"We are pleased to have this important recognition from the USPTO regarding our discovery of antagomirs, a new class of anti-miRs and a novel invention in the field of microRNA-based therapeutics," says Dr. Muthiah Manoharan, vice president for drug discovery at Alnylam. "With this initial grant, we have received broad claims covering classes of anti-miRs with certain important chemical features for optimal delivery and in vivo pharmacology. Importantly, the inventive nature of these novel compositions was allowed independent of any specific microRNA sequence, thereby providing a broad set of patent claims that add to the innovations Regulus can employ in designing novel microRNA therapeutics, an entire new class of medicines."  
"Regulus Therapeutics is advancing a whole new frontier of pharmaceutical research through the discovery of microRNA therapeutics, which have the potential to block broad disease pathways as opposed to a single target," says Dr. Kleanthis G. Xanthopoulos, president and CEO of Regulus Therapeutics. "Our access to the pioneering research of our academic collaborators and Alnylam and Isis on oligonucleotide-based therapeutics, as evidenced most recently by the award of the Manoharan patent, has enabled Regulus to build a dominant intellectual property estate for microRNA therapeutics. We believe this is a strong foundation to advance our innovative approach to developing novel medicines to address the needs of patients."
Code: E090921



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